LOS ANGELES, Calif. (Ivanhoe Newswire) – You heard it takes a village to raise a child. Well, in one little girl’s case, it took the right team of doctors, at the right time, with the right treatment to save her life.
Four-year-old Michelle Lowry is all about learning her letters.
And her numbers, but her parents didn’t know if they could count on seeing this day. What they thought was a tiny bug bite on Michelle’s neck turned out to be a tumor.
“It’s just pure terror and fear and the unknown,” Joe Lowry said.
After a battery of tests … doctors at Children’s Hospital Los Angeles diagnosed Michelle with a soft tissue sarcoma.
“It was all the way almost around her neck,” Christina Lowry described.
Because of the location, surgery and radiation were ruled out. Doctors used a new precise cancer panel called OncoKids to read Michelle’s DNA and RNA. The panel revealed Michelle would be a perfect candidate for a targeted new drug, Larotrectinib. Doctors worked fast to get Michelle in a new clinical trial.
“She was getting worse by the moment,” Christina Lowry said.
An investigational pharmacist raced in to prepare the twice-daily doses. And within 24 hours of her first dose, Michelle’s tumor had shrunk.
Jaclyn Biegel, PhD, FACMG, director of the Center for Personalized Medicine at Children’s Hospital Los Angeles said, “She went from being in really extreme situation to out of the ICU and off the floor and home in a couple of days.”
Two months later, her tumor had shrunk by 60 percent. Two years later, the tumor is undetectable.
“We look at her and we think we’re lucky; we’re blessed,” Joe Lowry said.
Blessed for the oncologists, radiologists, pathologists, pharmacists and lab techs who saved this little girl’s life.
Based on other positive outcomes like Michelle’s, the FDA granted accelerated approval of Larotrectinib. OncoKids is the first cancer panel designed specifically for children’s cancers and can detect genetic alterations for pediatric cancers including leukemias, solid tumors, sarcomas and brain tumors.
Contributors to this news report include: Marsha Lewis, Producer; Rusty Reed, Videographer; Ken Ashe, Editor.
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MEDICAL BREAKTHROUGHS
RESEARCH SUMMARY
TOPIC: THE RIGHT TEAM, TIMING AND TREATMENT SAVE MICHELLE
REPORT: MB #4715
BACKGROUND: Childhood soft tissue sarcoma occurs when cancer cells form in soft tissues of the body. Though it occurs in adults as well, having certain diseases and inherited disorders can increase the risk of it occurring in childhood. The most common sign of childhood soft tissue sarcoma is a painless lump or swelling in soft tissue of the body. If tests show there may be a soft tissue sarcoma, a biopsy is done. There are many classifications such as fat tissue tumor, skeletal muscle tumor, nerve sheath tumor, etc. Certain factors affect available treatment options and the patients’ chance of recovery. Diagnostic tests include physical exam, X-rays, MRI or magnetic resonance imaging, CAT scan, and/or ultrasound exam. Treatment options will vary and may cause side effects. Standard treatment includes but is not limited to drug therapy, surgery, chemotherapy, targeted therapy, immunotherapy, and observation.
(Source: https://www.cancer.gov/types/soft-tissue-sarcoma/patient/child-soft-tissue-treatment-pdq)
“In children the tumors can arise in different parts of the body and they’re diagnosed based on what the cells look like and what the genetic alterations are. That’s true in adults as well, but the primary diagnosis for adults are based on the anatomic location – so breast cancer, prostate cancer, lung cancer, colon cancer. Children don’t typically get those cancers, those are rare, and those are what we call epithelial tumors. The changes that you see in those tumors as well as the drugs that you use to treat them are different. Children are not just little adults; they actually have different types of cancer. For example, they can withstand a lot more chemotherapy than adults can. They respond better to bone marrow transplant, they recover better, and so you have to take everything into account.” (Jaclyn A Biegel, PhD, FACMG)
ONCOKIDS: OncoKids is a targeted, next generation sequencing panel that includes both RNA and DNA content. It is used to diagnose children with all types of cancer, from leukemia to brain tumors and solid tumors. The test helps determine prognosis and potentially identify therapies for the patient. It is more comprehensive than the previous adult-focused panel, including content using these DNA and RNA targets for pediatric cancer, which develops differently from adult cancers. (Jaclyn A Biegel, PhD, FACMG)
LAROTRECTINIB: Larotrectinib is the second drug after Merck’s Keytruda to be approved as a “tissue-agnostic” drug by the FDA. This means it is not directed at cancers in specific organs, but rather at the cancers caused by particular genetic mutations (ideal for childhood soft tissue sarcomas.) Patients selected for consideration of this treatment are based on the presence of NTRK gene fusion in the tumor specimens. In clinical trials, identifying this positive gene fusion status was determined in local labs using NGS or fluorescence testing. The recommended dose is orally twice daily with food, dose size varying to the patient. Clinical trials evaluated both adults and pediatric patients with these tumors, and overall response rates were positive.
(Sources: https://www.acs.org/content/acs/en/molecule-of-the-week/archive/l/larotrectinib.html
FOR MORE INFORMATION ON THIS REPORT, PLEASE CONTACT:
Lauren Song
lasong@chla.usc.edu
If this story or any other Ivanhoe story has impacted your life or prompted you or someone you know to seek or change treatments, please let us know by contacting Marjorie Bekaert Thomas at mthomas@ivanhoe.com
